FDA Approves First Gene Therapy For An Inherited Disease

In a historic move, the Food and Drug Administration on Tuesday approved a pioneering gene therapy for a rare form of childhood blindness, the first such treatment cleared in the United States for an inherited disease.

The approval signals a new era for the gene therapy field, which struggled for decades to overcome devastating setbacks but now is pushing forward in an effort to develop treatments for everything from hemophilia to sickle-cell anemia. Yet the products, should they reach patients, are likely to carry stratospheric prices – a prospect already worrying consumer advocates and economists.

Philadelphia-based Spark Therapeutics, which makes the blindness treatment, said it will not announce the price until January. Analysts speculate it could be as much as $1 million for both eyes.